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Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)

Alpha 1-Antitrypsin Deficiency Clinical Trial

Official title:
A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)

Clinical Trial Summary

The purpose of this study is to evaluate the the safety and efficacy of the investigational product, fazirsiran (TAK-999, ARO-AAT), administered subcutaneously to patients with Alpha-1 Antitrypsin Deficiency.

Clinical Trial Description

Participants will be enrolled to receive multiple subcutaneous injections of fazirsiran (TAK-999, ARO-AAT). All eligible participants will require a pre-dose biopsy completed as part of the study within the screening window. All participants will undergo an End of Study (EOS) biopsy. Treated participants will be offered the opportunity to continue treatment in an open label extension during which they will undergo a final biopsy. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03946449
Study type Interventional
Source Arrowhead Pharmaceuticals
Contact
Status Active, not recruiting
Phase Phase 2
Start date December 19, 2019
Completion date August 23, 2024
View Details
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